House Subcommittee Presses Forward on 21st Century Cures

WASHINGTON -- Roughly 95% of rare diseases have no FDA-approved treatment. Congress hopes that by equipping government agencies with better resources, it can accelerate innovation and bring cures to patients faster.

The latest draft of the 21st Century Cures bill would grant $10 billion in mandatory funding to the National Institutes of Health beginning in fiscal year 2016.

"The research committee is ecstatic to see this new provision in the bill, and we are deeply appreciative," said Kathy Hudson, PhD, deputy director for Science, Outreach, and Policy at the NIH.

The House Energy and Commerce's Subcommittee on Health invited top brass from the NIH and the FDA to offer their perspectives of the revised legislation at a hearing on Thursday.

The five-member bipartisan team noted in a press release, "We've done things differently with 21st Century Cures, taking our time to listen and solicit feedback from every corner of the healthcare innovation infrastructure." They pointed to the 24 roundtable discussions across the country, eight hearings, and several white papers that preceded the hearing.

Rep. Joseph Pitts, (R-Pa.), chairman of the Subcommittee on Health, said, "While increasing accountability, this legislation would invest in the basic research so critical to equipping our nation's best and brightest with the tools they need to discover the underpinnings of disease."

In addition to funding NIH, provisions of the bill also support efforts to:

Speed development for treatment of life-threatening illness

Repurpose drugs found ineffective for one condition and test them in another

Develop partnerships among patients, providers, and researchers

Promote an interoperable health system

Enhance telehealth practices

Advance the development of more targeted personalized treatments

Other provisions in the bill explored ways to bring patients' perspectives into the research process.

Janet Woodcock, MD, director of the FDA's Center for Drug Evaluation and Research, spoke of 20 "facilitated discussions" with patients around the burden of their disease and the effects of treatment. Research teams documented the conversations in a report called "The Voice of the Patient" and on occasion issued guidance based on patient feedback.

"What we learned is we need a much more structured and organized way to incorporate this input into drug development, and we think that what's laid out in the discussion drafts will really help with that," Woodcock said.

Other representatives spoke about the importance of having interoperability among electronic health records. Rep. Michael Burgess ( R-Texas) was frustrated that $28 billion had been sunk into trying to make electronic health records talk to each other, but "health data continues to be fragmented."

Rep. Pitts added, "Imagine a world where your cellphone would not work with a land-line or if my cellphone did not connect with other networks. Ridiculous. Well, that's the world of electronic health records."

Hudson sympathized. In moving her mother from Texas to Minnesota she said, "I ended up carrying two boxes of paper medical records with me."

She added,"I hope that doesn't happen in the future, and I think we're moving quickly to solve that problem."

Hudson noted that interoperable systems are important in patient care but also "vital" for research, and especially important as the Precision Medicine initiative takes shape.

The Public Responds

Mary Woolley, president of Research!America, a nonprofit focused on public education, was pleased to see Upton and DeGette keep their promise to fund medical research.

"While it is important for the final language in the bill to allow for flexibility in the use of these funds in order to maximize their benefit, these additional dollars can empower NIH to sustain and embark on innovative studies that could reduce the prevalence and impact of costly and disabling conditions that continue to threaten individual and population health, our economic security, and global competitiveness," said Woolley in a press release.

Vijay Das, policy advocate at Public Citizen's Congress Watch, was less enthusiastic.

Das, in a press release, said that the new proposal is "riddled with dangerous provisions that would put patients at risk."

"The 21st Century Cures initiative offers a horse trade: Increase funding for the world-renowned National Institutes of Health (NIH) in exchange for providing perks to the pharmaceutical and medical device industries to approve medications and devices faster based on weaker evidence," Das said.

"Faster approval of new medicines will not spur innovation, but it will sacrifice safety. Passage of this legislation would put lives at risk."

Woodcock told MedPage Today that while the new bill would speed development to biomarkers "[t]here's no change to the time of our review process."

"Most of the provisions here also are not for common diseases that have a lot of alternative therapies. They're going to be for cancers or rare serious disease where the people have already said they're willing to trade off some uncertainty to get access to treatment sooner."

"I think it's good of the patient groups to remind people that [safety is] an important side of the equation," Woodcock added. "We don't see anything in here that's going to diminish that."

Full Steam Ahead

Rep. DeGette said she found the level of consensus around the bill "striking and positive."

She acknowledged that several members had commented on the new burdens the FDA would be tasked with as a result of provisions in the bill. Rep. Pallone counted more than 15 new guidance documents as well as separate approval processes for antibacterial and antifungal drugs for serious and life-threatening diseases.

Woodcock made clear that fulfilling these statutory requirements would create delays in reviewing applications, if the FDA was not given additional resources. "Currently, our drug review program is really going full speed. We're making all our deadlines and we'd like to keep it that way."

DeGette said, "We still need to find a way to fund the FDA for the things that we're asking you to do and we know that. So, we're going to do all of that."

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